CHICAGO – The opening of the 2013 Biotechnology Industry Organization's (BIO) annual International Convention in Chicago not only marks a celebration of the event's 20th anniversary but also is the second return to the Windy City during the past three years. When BIO 2010 convened here at the beginning of May the biotechnology industry was still reeling from the unprecedented global economic perturbations that tanked the capital markets in 2009. However, there was some promising news to help

"We know that patient foundations, historically, have driven research and evaluation of potential therapeutic approaches in rare diseases, and pharma has joined them more recently, with the exception of Genzyme [Corp.], of course, which has been there since the very beginning." – Cristina Csimma, CEO, Cydan LLC "We're at a very interesting moment in time in the antibody drug conjugate space. The older technology wasn't the most robust, so there were some failures preclinically and clinically

The Arkansas Supreme Court is being asked to overturn the $1.2 billion penalty assessed against Janssen Pharmaceuticals Inc. in a case that could open other drugmakers to hefty penalties when the state decides FDA-approved labeling is inadequate. The lawsuit, filed by the state of Arkansas, stemmed from the placement of risk information on the FDA-approved label for Risperdal (risperidone). Concerned that uninsured patients are not getting the intended benefit of the federal 340B drug discount

Company (Location) Product Description Indication Status Date Actavis Inc. (Parsippany, N.J.) Fortesta Testosterone 2% gel Deficiency or absence of endogenous testosterone Filed an abbreviated NDA 3/4/13 Astellas Pharma Inc. (Tokyo) Myrbetriq Mirabegron, extended-release tablets Overactive bladder Won Health Canada approval 3/11/13 Boehringer Ingelheim GmbH (Ingelheim, Germany) and Eli Lilly and Co. (Indianapolis) Empagliflozin Sodium glucose co-transporter-2 inhibitor Type II diabetes

Company (Location) Product Description Indication Status Date AUTOIMMUNE Biogen Idec Inc. (Weston, Mass.) Tecfidera Dimethyl fumarate Multiple sclerosis Was approved by the FDA 3/28/13 CANCER Acceleron Pharma Inc. (Cambridge, Mass.) ACE-536 A protein therapeutic that increases red blood cells Beta-thalassemia and myelodysplastic syndromes FDA granted orphan designation 3/27/13 A.P. Pharma Inc. (Redwood City, Calif.) APF530 A long-acting formulation of granisetron To prevent chemotherapy-induced

Company (Location) Product Description Indication Status Date AUTOIMMUNE Trophos SA (Marseille, France) Olesoxime A mitochondrial-targeted, cholesterol-like compound Multiple sclerosis Started a Phase Ib study 3/15/13 CANCER Anaeropharma Science Inc. (Tokyo) APS001F A living recombinant bifidobacterium Advanced or metastatic solid tumors Started a Phase I trial 3/29/13 Bio-Path Holdings Inc. (Houston) BP-100-1.01 Liposomal Grb-2 Blood cancers Started enrolling patients in the fifth dosage

Financings Anthera Pharmaceuticals Inc. secured a $20 million debt financing facility through a syndicate. Chimerix Inc. priced its initial public offering (IPO) of 7.3 million shares at $14 each, at the midpoint of its proposed range. Cleave Biosciences Inc. raised $10 million in an extension of its Series A financing. Coronado Biosciences Inc. netted $9.9 million through its at-the-market program. GenSight Biologics raised $41.6 million in a Series A funding. MedDay raised $10.5 million to

Resverlogix Corp. announced last week that it's spinning out its epigenetics platform into RVX Therapeutics Inc., leaving its ApoA-I stimulating drug candidate, RVX-208, with the parent company. The plan has been in the works for eight years since RVX Therapeutics was established as a subsidiary, Donald McCaffrey, president and CEO of Resverlogix, told BioWorld Insight. "We always felt that if the lead drug was of interest to someone and acquired we really didn't want to give up all the other

Company (Location) Product Description Indication Status Date Kythera Biopharma-ceuticals Inc. (Calabasas, Calif.) ATX-101 Injectable drug To reduce submental fat Phase IIIb data showed it is well tolerated and may be effective 3/5/13 Merz North America (Greensboro, N.C.) Naftifine hydrochloride Gel 2% Tinea pedis Company reported the ability of the gel to be both efficacious and tolerable in the treatment for interdigital-type and moccasin-type Tinea pedis 3/5/13 Sanofi SA (Paris) and

Company (Location) Product Description Indication Status Date AUTOIMMUNE Halozyme Therapeutics Inc. (San Diego) and Baxter International Inc. (London) HyQvia Combination of human normal immunoglobulin and recombinant human hyaluronidase designed as a replacement therapy Primary and secondary immunodeficiencies Received a positive opinion from the EMA's CHMP 3/25/13 Biogen Idec Inc. (Weston, Mass.) Tecfidera Dimethyl fumarate Relapsing-remitting multiple sclerosis Received a positive opinion

Company (Location) Product Description Indication Status Date AUTOIMMUNE Anacor Pharmaceuticals Inc. (Palo Alto, Calif.) AN2728 Topical boron-based phosphodiesterase-4 inhibitor Mild to moderate atopic dermatitis Phase II data showed it achieved a 71% improvement from baseline 3/22/13 GeNeuro SA (Geneva) GNbAC1 A humanized monoclonal antibody targeting a human endogenous retrovirus Multiple sclerosis Completed a Phase IIa study 3/15/13 CANCER Biothera Inc. (Eagen, Minn.) Imprime PGG Immuno

Company (Location) Product Description Indication Status Date AUTOIMMUNE Anthera Pharmaceuticals Inc. (Hayward, Calif.) Blisibimod A peptibody antagonist of BAFF Lupus Started a Phase III trial 3/29/13 Biogen Idec Inc. (Weston, Mass.) Plegridy Peginterferon beta-1a Relapsing-remitting multiple sclerosis Two-year Phase III data showed it significantly reduced MS disease activity, including relapses, disability progression and brain lesions, compared to placebo at one year 3/21/13 Celgene Corp

Company (Location) Product Description Indication Status Date Acadia Pharmaceuticals Inc. (San Diego) Pimavanserin The nondopaminergic blocker of serotonin 5-HT2A receptors Parkinson's disease psychosis Pivotal Phase III data demonstrated a highly significant antipsychotic efficacy on the nine-item SAPS-PD scale; the drug also met a key secondary endpoint 3/22/13 Biotie Therapies Corp. (Turku, Finland) SYN115 Tozadenant Parkinson's disease Phase IIb data showed significant reductions in mean

Like the number of biotech initial public offerings (IPOs) that are completed annually, another closely tracked statistic is the transactional value of the industry's mergers and acquisitions (M&As) and partnering deal flow. The number of deals concluded each year provides a measure of the vibrancy of the sector, its business strategies and technology aspirations. Over the past several years we have become used to seeing the completion of several multi-billion dollar M&A deals involving big

WASHINGTON – At the annual meeting of the American Association for Cancer Research (AACR) last week, researchers were touting the potential of immunotherapies at the same time as they were trying to figure out how to meet their unique regulatory and developmental challenges. And at the opening press conference, where scientists announced the results of several clinical trials of immunotherapies, discussant Louis Weiner, of Washington University in St. Louis, went so far as to suggest that